Article
Rescue of hearing by adenine base editing in a humanized mouse model of Usher syndrome type 1F.
Molecular therapy : the journal of the American Society of Gene Therapy - 2 Aug 2023
Peters Cole W, Hanlon Killian S, Ivanchenko Maryna V, Zinn Eric, Linarte Elizabeth F, Li Yaqiao, Levy Jonathan M, Liu David R, Kleinstiver Benjamin P, Indzhykulian Artur A, Corey David P
Abstract excerpt
Usher syndrome type 1F (USH1F), characterized by congenital lack of hearing and balance and progressive loss of vision, is caused by mutations in the PCDH15 gene. In the Ashkenazi population, a recessive truncation mutation accounts for a large proportion of USH1F cases. The truncation is caused by a single C→T mutation, which converts an arginine codon to a stop (R245X). To test the potential for base editors to...
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